KR20240023630A - 신규 rna 치료제 및 그의 용도 - Google Patents

신규 rna 치료제 및 그의 용도 Download PDF

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Publication number
KR20240023630A
KR20240023630A KR1020247002170A KR20247002170A KR20240023630A KR 20240023630 A KR20240023630 A KR 20240023630A KR 1020247002170 A KR1020247002170 A KR 1020247002170A KR 20247002170 A KR20247002170 A KR 20247002170A KR 20240023630 A KR20240023630 A KR 20240023630A
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South Korea
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sequence
seq
set forth
misc
feature
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KR1020247002170A
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Korean (ko)
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멜리사 앤 벨린저
토마스 패트릭 베이어
크리스틴 치-타오 청
메리진 에겐
그레고리 로렌스 라크너
레베카 루스 마일스
지보 왕
패트릭 조셉 안토넬리스
타카코 윌슨
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일라이 릴리 앤드 캄파니
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Publication of KR20240023630A publication Critical patent/KR20240023630A/ko

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/545—Heterocyclic compounds
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P3/06—Antihyperlipidemics
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
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  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247002170A 2021-06-24 2022-06-22 신규 rna 치료제 및 그의 용도 KR20240023630A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163214584P 2021-06-24 2021-06-24
US202163214555P 2021-06-24 2021-06-24
US63/214,555 2021-06-24
US63/214,584 2021-06-24
PCT/US2022/034504 WO2022271808A1 (en) 2021-06-24 2022-06-22 Novel rna therapeutics and uses thereof

Publications (1)

Publication Number Publication Date
KR20240023630A true KR20240023630A (ko) 2024-02-22

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ID=82608081

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Application Number Title Priority Date Filing Date
KR1020247002170A KR20240023630A (ko) 2021-06-24 2022-06-22 신규 rna 치료제 및 그의 용도

Country Status (11)

Country Link
US (1) US20230022590A1 (es)
EP (1) EP4359530A1 (es)
KR (1) KR20240023630A (es)
AU (1) AU2022297443A1 (es)
CA (1) CA3224145A1 (es)
CO (1) CO2023018345A2 (es)
DO (1) DOP2023000283A (es)
EC (1) ECSP23096460A (es)
IL (1) IL309411A (es)
TW (1) TW202315646A (es)
WO (1) WO2022271808A1 (es)

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
EP2992009B1 (en) * 2013-05-01 2020-06-24 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating apolipoprotein (a) expression
KR102493872B1 (ko) 2016-09-02 2023-01-30 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
US20220025367A1 (en) 2018-11-23 2022-01-27 Sanofi Novel rna compositions and methods for inhibiting angptl8

Also Published As

Publication number Publication date
WO2022271808A1 (en) 2022-12-29
US20230022590A1 (en) 2023-01-26
ECSP23096460A (es) 2024-01-31
CO2023018345A2 (es) 2024-05-10
TW202315646A (zh) 2023-04-16
AU2022297443A1 (en) 2024-01-04
IL309411A (en) 2024-02-01
CA3224145A1 (en) 2022-12-29
DOP2023000283A (es) 2024-02-29
EP4359530A1 (en) 2024-05-01

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